Rare cranial disorders: Towards a non-invasive therapy using gene silencing delivered by nanoparticles and 3D printing

A “gene silencer” (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an injectable gel produced through 3D printing, can switch off the defective gene responsible for serious rare diseases known as craniosynostoses. These conditions are characterized by malformations of the skull and are currently treatable only through highly invasive surgical procedures performed in newborns and often repeated throughout childhood.

This article was originally published on MedicalXpress.com

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